Cartherics' AU$15M series B advances CTH-401 for ovarian cancer
Immunotherapy company Cartherics Pty Ltd. raised AU$15 million (US$10.3 million) in an oversubscribed series B round that will support the first clinical trial for lead chimeric antigen receptor natural killer therapy CTH-401 for ovarian cancer, and to expand its pipeline to include other diseases. Cartherics CEO Alan Trounson told BioWorld that the funds raised will take Cartherics through to mid-2026, and the phase I Australian trial in ovarian cancer will begin in the fourth quarter of 2025.

Cartherics' AU$15M series B advances CTH-401 for ovarian cancer
Immunotherapy company Cartherics Pty Ltd. raised AU$15 million (US$10.3 million) in an oversubscribed series B round that will support the first clinical trial for lead chimeric antigen receptor natural killer therapy CTH-401 for ovarian cancer, and to expand its pipeline to include other diseases. Cartherics CEO Alan Trounson told BioWorld that the funds raised will take Cartherics through to mid-2026, and the phase I Australian trial in ovarian cancer will begin in the fourth quarter of 2025.
MTA-cooperative PRMT5 inhibitor exerts robust antitumor activity
The gene encoding methylthioadenosine phosphorylase (MTAP) is expressed in normal tissues but 10 to 15% of tumors present deletions of MTAP expression that lead to accumulation of its metabolite 5-methylthioadenosine (MTA). Previous research has shown that selective inhibition of protein arginine methyltransferase 5 (PRMT5) in the presence of MTA may be considered a potential therapeutic strategy for the treatment of MTAP-deleted cancer.
DNA vaccine pVAX1-SRS13 protects against chronic toxoplasmosis in vivo
Ege University scientists and their collaborators have detailed the preclinical characterization of a novel DNA vaccine encoding the SRS13 protein being developed for the prevention of chronic toxoplasmosis.
Other news to note for Oct. 3, 2024
Additional early-stage research and drug discovery news in brief.
US scientists patent ER-β ligands for endometriosis and multiple sclerosis
Scientists at University of California and University of Illinois have disclosed compounds acting as estrogen receptor β (ER-β; ESR2) ligands.
Alentis Therapeutics’ anti-CLDN1 ADC gains IND clearance
Alentis Therapeutics AG has obtained IND clearance from the FDA for ALE.P02, an anti-Claudin-1 (CLDN1) antibody-drug conjugate (ADC) with a tubulin inhibitor payload.
Preclinical profiling of Splisense’s inhaled antisense oligonucleotide
Researchers from Splisense Ltd. and affiliated organizations recently reported preclinical data for SPL-84, an inhaled antisense oligonucleotide drug candidate being developed for the treatment of patients with cystic fibrosis carrying the 3849 +10 kb C-to-T (3849) mutation.
Protein kinase inhibitors patented by Baylor College of Medicine and Deliver Therapeutics
Researchers at Baylor College of Medicine and Deliver Therapeutics Inc. have synthesized protein kinase inhibitors reported to be useful for the treatment of cancer and psoriasis.

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Conferences

PDE4 inhibitor ME-3183 shows promising preclinical efficacy in atopic dermatitis
Dermatologic
Phosphodiesterase 4 (PDE4) is an enzyme known to play a role in inflammatory responses, and it has been pinpointed as an interesting therapeutic target for diseases like atopic dermatitis (AD), but limited efficacy and side effects have prevented the approval of oral formulations.
FABP5 inhibitor ART-26.12 exhibits activity in psoriasis models
Dermatologic
Artelo Biosciences Inc. has presented data on its fatty acid-binding protein 5 (FABP5) inhibitor ART-26.12 from testing of its efficacy in preclinical models of psoriasis.
4D-710 gene therapy helps overcome lack of response to CFTR modulators
Genetic/congenital
Scientists from 4D Molecular Therapeutics Inc. disclosed the preclinical evaluation of 4D-710, an aerosolized gene therapy that consists of a lung-specific evolved A101 capsid vector, the promoter CMV173 and the transgene codon-optimized human CFTRΔR.
CSNK1D variant tied to sleep-wake phase syndrome
Biomarkers
Italian researchers have presented a case study of pediatric advanced sleep-wake phase syndrome in a 4-year-old girl. Autosomal dominant forms of this syndrome due to loss-of-function mutations in the gene coding casein kinase I isoform delta (CSNK1D) have previously been described.

Cancer

Prime’s $3.61B Bristol Myers deal is year’s fourth largest
Deals and M&A
In the year’s fourth-largest deal, Prime Medicine Inc. will collaborate with Bristol Myers Squibb Co. in a research collaboration and license agreement totaling $3.61 billion. The two companies plan to develop reagents for ex vivo T-cell therapies. While the programs and targets have yet to be...

Infection

Discovery of macrocyclic 3CLpro inhibitors with improved PK properties
Coronavirus
In work at Shanghai Curegene Pharmaceutical Co. Ltd., synthesis and optimization of a series of SARS-CoV-2 3CL protease (3CLpro, Mpro) inhibitors led to the identification of compounds [I], [II] and [III] as lead candidates suitable for further evaluation, based on their enzymatic IC50s (14, 12 and...

Precision Biosciences files CTAs for PBGENE-HBV for chronic HBV
Regulatory
Precision Biosciences Inc. has submitted its first clinical trial applications (CTAs) to initiate a phase I study evaluating PBGENE-HBV, an in vivo gene editing program designed to potentially cure chronic hepatitis B virus (HBV).
Phylogeographics, phage typing combo fights antibiotic resistance
The spread of drug-resistant bacteria is a global health concern and could once again become a leading cause of mortality. The World Health Organization has flagged carbapenem-resistant Acinetobacter baumannii (CRAB), which has a mortality rate of 25%-60%, as a top priority pathogen requiring...

Neurology/psychiatric

140,000 cells and 50M synapses make 1 adult fly brain
Science
A collaboration led by the Flywire Consortium and comprising hundreds of scientists has completed a whole map of the adult fruit fly brain after several decades of collaborative work. By using electron microscopy and three-dimensional reconstruction supported by AI tools, the researchers have...
Loqus23 Therapeutics to advance somatic expansion inhibitors
Financings
Loqus23 Therapeutics Ltd. has closed a £35 million (US$43 million) series A financing, with the aim of supporting its work developing small-molecule somatic expansion inhibitors for the treatment of Huntington’s disease and other triplet repeat disorders.
In Alzheimer’s, risk gene combination affects females more
The E4 variant of the APO gene, the R47H variant of the TREM2 gene, and female sex are three of the strongest risk factors for the development of Alzheimer’s disease (AD). By combining all three of them in a mouse model of tauopathy, researchers at Weill Cornell Medical School have identified...
MYC inhibition contributes to multiple sclerosis amelioration
Conferences
MYC is a transcription factor that plays relevant roles in cellular processes such as glycolysis, development, cell differentiation or proliferation. Recent research has associated the transcriptional activity of MYC to the activation of T cells in multiple sclerosis.
HDAC3 inhibition counteracts autoimmune demyelination in mice
Conferences
After a team at the Hospital Universitari Vall d’Hebron found that histone deacetylase 3 (HDAC3) has a role in the chronic inflammatory response in the central nervous system in models of traumatic brain injury, they investigated its role in autoimmune demyelination.

Endocrine/metabolic

Papillon’s PPL-002 awarded US orphan drug designation for Danon disease
Regulatory
The FDA has awarded orphan drug designation to Papillon Therapeutics Inc.’s PPL-002, an experimental gene-modified CD34+ hematopoietic stem and progenitor cell (HSPC) therapy, for the treatment of Danon disease.

Biomarkers

RAC1 has prognostic value in hepatocellular carcinoma
Cancer
Hepatocellular carcinoma (HCC) has a poor prognosis due to late diagnosis and lack of effective therapies.

Variant in ITPR3 behind a complex multisystemic disorder
Neurology/psychiatric
The homeostasis of cytosolic calcium (Ca2+) is crucial for several physiological functions such as cell motility and neuronal transmission, among others. In this process, the inositol 1,4,5-trisphosphate receptor type 3 (ITPR3) plays a key role in the release of intracellular Ca2+.
ZFP1 is a prognostic marker in gastric cancer, study finds
Cancer
Gastric cancer (GC) is among the most malignant tumors in terms of incidence and mortality. Early diagnosis can improve the survival rate of patients, but there is a lack of effective biomarkers for the diagnosis and prognosis of GC. Zinc finger protein 1 homologue (ZFP1) plays an important role in...

Gastrointestinal

Shattuck Labs to focus on DR3 antagonist antibody for IBD
Antibody
Shattuck Labs Inc. has announced a strategic shift to focus on SL-325, a death receptor 3 (DR3) antagonist antibody for the treatment of inflammatory bowel disease (IBD) and other inflammatory autoimmune diseases.
Palisade submits CTA for PDE4 inhibitor prodrug for ulcerative colitis
Regulatory
Palisade Bio Inc. has submitted a clinical trial application (CTA) for PALI-2108 for the treatment of ulcerative colitis, following a pre-CTA consultation meeting with Health Canada. Pending clearance, the company anticipates initiating a phase I...
Grants support Mabylon’s programs for ALS and inflammatory bowel disease
Neurology/psychiatric
Mabylon AG has been awarded three grants totaling more than CHF1.3 million (US$1.5 million) from Innosuisse Swiss Innovation Agency, Target ALS and the ALS Association.
New CXXC5/DVL interaction inhibitors disclosed in CK Regeon patent
Patents
CK Regeon Inc. has divulged indirubin derivatives acting as CXXC-type zinc finger protein 5 (CXXC5)/DVL interaction inhibitors reported to be useful for the treatment of skin lesions, wounds, neurodegeneration, metabolic disorders and bone disorders.
Orbis to use Vivtex platform for gut permeability screening
Deals and M&A
Orbis Medicines SA has announced a research collaboration and option to an exclusive license with Vivtex Corp.
EASD 2024: Incretins play all the instruments in the orchestra
Conferences
Incretins are not just metabolic hormones that regulate glucose levels after eating. Their functions go beyond stimulating the release of insulin from the pancreas. Glucagon-like peptide-1 (GLP-1) and glucose-dependent insulinotropic polypeptide...

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